EMA clears gene therapy



The European Medicines Agency has given a conditional marketing authorisation to a gene therapy for haemophilia B which is a one-time treatment for the rare inherited blood disorder. Announced on 31 May, the decision is for fidanacogene elaparvovec, an adeno-associated virus based therapy that is designed to deliver a functional copy of the Factor 9 gene into the body enabling patients to produce Factor 9 protein themselves. The developer is Pfizer Inc which received US regulatory approval for the same therapy in late April.